A Prospective, One-arm and Open Clinical Study of CM313 in the Treatment of Immune Thrombocytopenia

NCT05694767 | Completed Phase 2 DMC

• 1 other identifier: IIT2022039（2）
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and efficacy of CM313 in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.

Conditions

Immune Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (2)

• To evaluate the efficacy after CM313 treatment within 8 weeks

  Proportion of subjects with a platelet count ≥ 50 × 10\^9/L within 8 weeks after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period

  8 weeks

• Safety of CM 313

  Incidence, severity, and relationship of treatment emergent adverse events after CM 313 treatment

  24 weeks

Secondary Outcomes (7)

• Other efficacy evaluation

  12 weeks

• Duration from treatment initiation to platelet count ≥30×10^9/L and ≥50×10^9/L

  12 weeks

• Cumulative weeks of platelet ≥30×10^9/L and platelet ≥50×10^9/L

  24 weeks

• Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale

  24 weeks

• Measurements of platelet glycoprotein (GP) autoantibodies

  24 weeks

Study Arms (1)

Intervention (CM313)

EXPERIMENTAL

20 enrolled subjects: once a week x 8 doses

Drug: CM313 Injection

Interventions

CM313 Injection DRUG

intravenous CM313 administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with CD38 monoclonal antibody (CM 313: 16mg/kg/w) for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg CM313 once a week for 8 weeks to observe the safety and efficacy during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of CM313 after treatment.

Intervention (CM313)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 and above, male or female
• Conform to the diagnostic criteria of immune Thrombocytopenia (ITP)
• Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab or TPORAs.
• The previous emergency treatment of ITP (e.g. methylprednisolone, platelet transfusion, IVIG transfusion) must be completed at least 2 weeks before the first administration
• Signed and dated written informed consent
• With normal hepatic and renal functions
• ECOG physical state score ≤ 2 points
• Cardiac function of the New York Society of Cardiac Function ≤ 2
• Patients receiving maintenance treatment (including corticosteroids (less than or equal to 0.5mg/kg prednisone), TPO receptor agonists, etc.) must have a stable dose at least 4 weeks before the first administration, and azathioprine, danazol, cyclosporin A, tacrolimus, sirolimus, etc. must be stopped at least 4 weeks before the first administration; The end of rituximab treatment was\>3 months；More than 6 months after splenectomy.
• April 10, 2023 After approval by the Ethics Committee on , subjects no longer require platelet glycoprotein autoantibodies positivity upon enrollment.

You may not qualify if:

• Received any treatment of anti-CD38 antibody drug
• Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
• HIV positive;
• Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
• Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
• At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
• Patients with thrombotic diseases such as pulmonary embolism, thrombosis and atherosclerosis;
• Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
• Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
• Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
• Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
• Patients with septicemia or other irregular severe bleeding;
• Patients taking antiplatelet drugs at the same time;
• Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

Location

Related Publications (1)

• Chen Y, Xu Y, Li H, Sun T, Cao X, Wang Y, Xue F, Liu W, Liu X, Dong H, Fu R, Dai X, Wang W, Ma Y, Song Z, Chi Y, Ju M, Gu W, Pei X, Yang R, Zhang L. A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia. N Engl J Med. 2024 Jun 20;390(23):2178-2190. doi: 10.1056/NEJMoa2400409.

  PMID: 38899695 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2022
• First Posted: January 23, 2023
• Study Start: January 22, 2023
• Primary Completion: December 30, 2023
• Study Completion: December 30, 2023
• Last Updated: February 24, 2025

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)